News Release | sareptatherapeutics.com

News Release | sareptatherapeutics.com: CAMBRIDGE, MA -- (Marketwired) -- 10/02/13 -- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced a new online resource center, called Let's Skip Ahead, for families affected by Duchenne muscular dystrophy (DMD) and their healthcare providers. The new website, available at www.skipahead.com, includes information and educational resources intended to make the science of exon skipping simple, and provides an opportunity to sign up for updates about upcoming Sarepta clinical trials.

http://www.skipahead.com/

News Release | sareptatherapeutics.com

News Release | sareptatherapeutics.com: Sarepta Therapeutics Announces Eteplirsen Demonstrates Continued Stability on Walking Test Through 96 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced data through Week 96 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results through nearly two years showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT). As previously reported, Study 202 met its primary endpoint of increased novel dystrophin as assessed by muscle biopsy at Week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes.

After 96 weeks, patients in the 30 mg/kg and 50 mg/kg eteplirsen cohorts who were able to perform the 6MWT (modified Intent-to-Treat or mITT population; n=6) experienced less than a 5 percent decline (17.5 meters) from baseline in walking ability. A statistically significant treatment benefit of 70.8 meters (p ≤0.001) was observed for the mITT population compared with the placebo/delayed-treatment cohort (n=4), which initiated treatment at Week 25 following 24 weeks of placebo. After experiencing a substantial decline earlier in the study, the placebo/delayed-treatment cohort also demonstrated stabilization in walking ability from Week 36 through 96, the period from which meaningful levels of dystrophin were likely produced, with a decline of 18.5 meters over this timeframe. These analyses were based on the maximum 6MWT score when the test was performed on two consecutive days.

"We are very encouraged the study has demonstrated walking stability in patients for more than a year since confirming that eteplirsen treatment produced dystrophin in their muscles," said Chris Garabedian, president and chief executive officer of Sarepta Therapeutics. "We look forward to sharing these updated data with the FDA as part of our New Drug Application for eteplirsen, which we plan to submit in the first half of 2014."

All Party Parliamentary Group - Access to high-cost drugs for rare diseases - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

All Party Parliamentary Group - Access to high-cost drugs for rare diseases - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Yesterday, Paul Lenihan – our CEO attended the Parliamentary reception briefing on the launch of the APPG’s report into access to high-cost drugs for rare diseases. Hosted and introduced by Dave Anderson MP, Action Duchenne endorses the four main recommendations contained within the report. These call for: 

1. The Government to establish ring-fenced funds for rare disease drugs. 

2. National Institute for Clinical Excellence (NICE) to access treatments for rare conditions in a different way from less rare conditions 

3. NICE and NHS England to speed up access to life-changing drugs after final stages of clinical trials to ensure there are no major delays in treatments reaching children. 

4. NHS England to ensure specialist centres are equipped with an appropriate range of health professionals to deliver treatments. 

Clinical trial recruitment updates - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Clinical trial recruitment updates - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: The FOR DMD study is now open to recruitment in most UK sites: Newcastle, GOSH, Cardiff, Manchester, Leeds, Liverpool, Glasgow and Birmingham.

The FOR DMD study is an international study which evaluate the three most commonly prescribed corticosteroid regimes in DMD to establish which one is of greatest benefit and which one is associated with the best side effect profile. Participating boys will receive one of the three regimes and will be closely monitored for muscle strength, performance and development of any side effects in accordance to the highest standards of care. Further information will de desiminated and please refer to the website for more details (link below). 

Recruitment for US phase Ib and IIa studies testing halofuginone (HT-100 by HALO Therapeutics) is currently exceeding targets. This compound is the first antifibrotic to be trialled in DMD and was previously supported at the early stages by various patients organisations, including Action Duchenne. Halofuginone, interrupts signalling in a key fibrotic pathway; preventing the replacement of normal muscle fibres and stabilising muscle function. 

The HALO team are planning ahead with larger multinational pivotal phase II/III studies estimated to take place in 2015 (see link at the end for further details). 

Clarity on Drisapersen breakthrough designation and Ataluren trial update - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

On the 28th of June, GSK announced that the US Food and Drug Administration (FDA) had awarded their drisapersen drug for exon 51 skipping breakthrough status. Having been in contact with the GSK team concerning it's significance, it is quite clear that the FDA will work closely with GSK to provide advice and guidance in order to review the drug in an efficient manner. Further information has also been taken from the FDA website, of which a link is included at the bottom of the page. 

PTC therapeutics have informed the Action Duchenne team that phase III trial sites will open this autumn in the UK. Their primary compound, Ataluren is a potential treatment for those with a nonsense mutation (about 13%) of the DMD population. They have produced a frequently asked question factsheet for those who would like further information - please follow the link below. 

http://www.actionduchenne.org/jsp/uploaded_files/documents/ROOT/2013-06-24%20FAQ%20FINAL%20for%20print%20ATA.pdf 


If there are any questions regarding DMD research or treatments on the horizon, please contact Diana Ribeiro, Head of Research: diana@actionduchenne.org. 

Experts and patient organisations unite to discuss DMD clinical trial parameters - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

On the 21st of June, Diana Ribeiro, Head of Research, Action Duchenne was involved in a meeting hosted by TREAT-NMD and co-sponsored by various MD organisations entitled Ways to Measure Clinical Effectiveness in the Investigation of Medicinal Products for BMD/DMD. This was a document drafted by the European Medicines Agency earlier this year (link below) and has gone out to consultation to all. They have invited further evidence-based comments and feedback in a coherent and structured manner by the 31st of August. 

The meeting led by the experts and family members pointed out areas they felt had not been clear with regards to current progress in assessing DMD clinical trials and based on current knowledge can be addressed further. A few representatives from the regulatory agencies were present and shared their personal views. Much of the discussion involved the robustness and validity of current and future outcomes for studies. 

There is more evidence to emerge for clinical trial criteria, which is currently unpublished to address some of the main limitations as stipulated by the respective agencies and regulators in the draft guideline document. These need to be assimilated by the experts into a formal response and the information from this meeting and consultation will be shared with all as soon as these become available.

Updates in exon skipping data - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy

Updates in exon skipping data - View News Article - Action Duchenne - Fighting for a cure for muscular dystrophy: Sarepta Therapeutics, Inc. announced updated data at the Wells Fargo Securities Healthcare Conference on the 19th of June from Study 202, a Phase IIb open-label extension study of eteplirsen for patients who have a genotype amenable to skipping of exon 51. Results at 84 weeks showed a continued stabilisation of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT).As previously reported, increased novel dystrophin was observed as assessed by muscle biopsy at week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes.